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Black Diamond Therapeutics Announces Strategic Priorities and Expected Milestones for 2022

On January 10, 2022 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a precision oncology medicine company pioneering the discovery and development of MasterKey therapies, reported a strategic pipeline update and outlined its expected upcoming milestones (Press release, Black Diamond Therapeutics, JAN 10, 2022, View Source [SID1234598508]).

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"MasterKey inhibitors target mutation families and are designed to address the unmet medical need of cancer patients with genetically defined cancers for whom precision therapies are not available," said David Epstein, Ph.D., Chief Executive Officer of Black Diamond Therapeutics. "We are incredibly pleased to have submitted the IND ahead of schedule for BDTX-1535, a next generation CNS penetrant MasterKey inhibitor designed to target EGFR driver mutations found in certain patients with GBM and NSCLC. The oncogenic alterations of EGFR, particularly those associated with GBM, result in distinct conformations which impart unique pharmacology and drug resistance. BDTX-1535 is designed to exploit this mechanism as a critical point of attack. We believe Black Diamond is uniquely positioned to deliver a pipeline of truly differentiated MasterKey programs as we leverage our expertise in cancer genomics, onco-protein function and drug discovery."

"As a result of the rapid evolution of the treatment landscape in NSCLC harboring EGFR or HER2 Exon 20 insertion mutations, we have decided to obtain further clinical data from the BDTX-189 safety expansion cohort at the recommended Phase 2 dose in 2022 in order to inform the future development of our BDTX-189 program, gating the start of a Phase 2 trial. The revised strategy enables near-term prioritization of BDTX-1535 clinical development and further investment in our pipeline, while allowing us to obtain more clinical data on BDTX-189, and simultaneously extends our cash runway into 2024."

Pipeline Updates and Expected Milestones:
BDTX-1535

Black Diamond announced ahead of schedule the submission of its Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for BDTX-1535 and expects to initiate the Phase 1 study of BDTX-1535 in the first quarter of 2022, subject to allowance of the IND by the FDA.
BDTX-1535 is designed as a potent, selective, brain-penetrant and irreversible MasterKey inhibitor of epidermal growth factor receptor (EGFR) mutations expressed in glioblastoma multiforme (GBM) and of intrinsic and acquired resistance EGFR mutations to third generation EGFR inhibitors in NSCLC.
In pre-clinical studies, Black Diamond has demonstrated that oncogenic alterations of EGFR, particularly those associated with GBM, result in distinct conformations which impart unique pharmacology and drug resistance. BDTX-1535 is designed to exploit this mechanism and has demonstrated anti-cancer activity and growth regressions across a panel of patient-derived xenograft models including intracranial tumor models.
It is estimated that approximately 50% of GBM patients harbor an oncogenic EGFR alteration that has the potential to be addressed by BDTX-1535, representing a potential patient population of greater than 60,000 patients annually across the US, EU, Japan and China.
It is estimated that across the US, EU, Japan and China there are approximately 20,000 patients who are diagnosed annually with non-small cell lung cancer (NSCLC) harboring an EGFR intrinsic or acquired resistance mutation.
The Company expects to provide a clinical data update on BDTX-1535 in the second half of 2023.
BDTX-189

BDTX-189 is designed as a MasterKey inhibitor targeting families of oncogenic mutations in EGFR and HER2.
Clinical data obtained from the MasterKey-01 study to date have demonstrated a favorable safety profile for BDTX-189 and early signs of clinical activity in patients whose tumors are driven by MasterKey mutation families, including two confirmed partial responses in heavily pretreated patients who have remained on treatment for more than 10 months.
Due to the rapid evolution of the treatment landscape in NSCLC harboring either EGFR or HER2 Exon 20 insertion mutations, the Company has decided to gate the initiation of the Phase 2 portion of the MasterKey-01 study and determine next steps in development based on further clinical data obtained from the safety expansion cohort at the recommended Phase 2 dose.
The Company expects to provide further guidance on the BDTX-189 program in 2022.
BRAF Program

Black Diamond is developing a CNS-penetrant BRAF inhibitor against a family of Class I, II, III canonical and non-canonical mutations. The Company’s lead BRAF compound is designed to be highly selective, potent and to avoid paradoxical activation.
In cell-based assays, Black Diamond’s lead BRAF compound demonstrated potent inhibition of a wide spectrum of BRAF mutations and fusions and exhibited dose-dependent inhibition of protein kinase RNA-like endoplasmic reticulum kinase downstream signaling.
In preclinical BRAF-driven tumor models, daily dosing of the lead compound demonstrated dose-dependent tumor growth inhibition, tumor regression and survival consistent with potent on-target and on-pathway inhibition.
The Company’s lead BRAF compound demonstrated robust brain penetration properties and achieved intracranial tumor growth inhibition in pre-clinical studies.
It is estimated that across the US, EU, and Japan there are approximately 190,000 patients with solid tumors who are diagnosed annually with BRAF oncogenic mutations.
Black Diamond anticipates the initiation of IND-enabling studies for the BRAF program in 2022.
MAP Discovery Engine

The Company continues to focus on building its Mutation-Allostery-Pharmacology (MAP) Discovery Engine to exploit mutant onco-protein conformations for the delivery of selective MasterKey therapies, with a focus on developing a MasterKey inhibitor that spares wild type fibroblast growth factor receptor 1 ( FGFR1) while inhibiting FGFR2 and FGFR3 mutant families, as well as other preclinical stage programs.
The Company plans to integrate new molecular dynamic modeling tools from its strategic partnership with OpenEye Scientific.
Financial Guidance

Black Diamond ended 2021 with approximately $210 million in cash, cash equivalents and investments, which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into 2024.

Karyopharm Announces Preliminary Unaudited Fourth Quarter and Full Year 2021 Net Product Revenues and Outlines 2022 Objectives

On January 10, 2022 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported preliminary unaudited fourth quarter and full year 2021 net product revenue estimates for XPOVIO, the Company’s first-in-class, oral XPO1 inhibitor, and outlined its 2021 achievements and 2022 objectives (Press release, Karyopharm, JAN 10, 2022, View Source [SID1234598507]).

Based on preliminary unaudited financial information, Karyopharm expects net product revenues of XPOVIO to be approximately $29.7 million for the fourth quarter and approximately $98.3 million for the full year 2021.

"For 2022, we are focused on making significant advances across our pipeline, beginning with top-line results from the Phase 3 SIENDO study evaluating selinexor as a maintenance therapy following front-line chemotherapy in patients with advanced or metastatic endometrial cancer, which remain on track to be reported this quarter," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "Building on the strong revenue growth in the second half of 2021, we will continue to prioritize driving sales and the adoption of XPOVIO in multiple myeloma. I am extremely pleased with the continued progress of our pipeline in key additional indications of multiple myeloma, myelodysplastic syndromes and myelofibrosis and our ability to further expand our impact on patients globally with our recent partnership with Menarini."

Key Program Achievements in 2021

Selinexor in Multiple Myeloma

Launched XPOVIO in the U.S. in second line multiple myeloma and strengthened the commercial team, resulting in continued strong net product revenue growth.
Generated compelling clinical data supporting selinexor’s efficacy, durability and tolerability when combined with the approved agents Velcade (bortezomib), Pomalyst (pomalidomide) or Kyprolis (carfilzomib) in patients who have been previously exposed to anti-CD38 monoclonal antibody treatment, where there is a substantial unmet need for a new class of therapy. These results were reported at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting, the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Annual Meeting and at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2021 Annual Meeting.
Multiple XPOVIO-based regimens are listed on the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines) for the treatment of second and later line multiple myeloma, including selinexor, pomalidomide and dexamethasone (SPd), which is an all-oral regimen, selinexor, daratumumab and dexamethasone (SDd), and selinexor, carfilzomib and dexamethasone (SKd).
The European Commission (EC) and the United Kingdom’s Medicines & Healthcare Products Regulatory Agency (MHRA) both granted conditional marketing authorization for NEXPOVIO (selinexor) for the treatment of relapsed or refractory multiple myeloma in patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.
Karyopharm’s Marketing Authorization Application (MAA) for NEXPOVIO in combination with Velcade (bortezomib) and low-dose dexamethasone for the treatment of multiple myeloma following at least one prior therapy has been validated by the European Medicines Agency (EMA) and is currently under review by the Committee for Medicinal Products for Human Use (CHMP).
Karyopharm’s global partners Antengene and Promedico, a member of the Neopharm Group, obtained approvals and conditional approvals for XPOVIO in multiple new geographies and indications, including in China, South Korea and Israel in penta-refractory multiple myeloma and relapsed and refractory diffuse large B-cell lymphoma (DLBCL).
Earned $10.0 million in milestone payments from Antengene in the third quarter of 2021 following the July 2021 approval of selinexor in South Korea for the treatment of patients with multiple myeloma and DLBCL.
Selinexor in Endometrial Cancer

Completed recruitment in the Phase 3 SIENDO study evaluating the efficacy and safety for front-line maintenance therapy with selinexor in patients with advanced or recurrent endometrial cancer.
Eltanexor in Myelodysplastic Syndromes (MDS)

Reported positive data from an investigator-sponsored Phase 1 study evaluating single-agent eltanexor in patients with hypomethylating agent (HMA)-refractory MDS, where eltanexor demonstrated a 53% overall response rate and median overall survival of 9.9 months. This compares favorably to historical survival of four to six months for HMA-refractory MDS patients.
Commenced dosing in a company-sponsored Phase 2 study evaluating single-agent eltanexor in patients with intermediate or high-risk HMA-refractory MDS.
Selinexor in Myelofibrosis (MF)

Reported positive data from an investigator-sponsored Phase 2 study evaluating single-agent selinexor in patients with MF previously treated with JAK inhibition. In this study, 40% of patients who received at least 24 weeks of selinexor treatment achieved a response, defined as ≥35% spleen volume reduction. Responses were durable with a median treatment duration of 11 months.
Commenced dosing in a company-sponsored Phase 2 study evaluating single-agent selinexor versus physician’s choice in patients with MF previously treated with a JAK 1/2 inhibitor.
Commenced dosing in a company-sponsored Phase 1/2 study evaluating selinexor in combination with Jakafi (ruxolitinib) in patients with treatment-naïve MF.
Corporate and Financial Highlights

Entered into an exclusive license agreement with Menarini Group to commercialize NEXPOVIO in Europe, Latin America and other key global territories; received $75 million upfront and eligible to receive up to $202.5 million in future milestones, plus tiered, double-digit royalties on net sales.
Received $60 million in expanded royalty agreement with entities managed by HealthCare Royalty Management, LLC, with up to another $40 million in potential financing available.
Anticipated 2022 Catalysts and Operational Objectives

Leverage commercial capabilities and increase U.S. XPOVIO sales in 2022.
Report top-line data from Phase 3 SIENDO study evaluating selinexor as a maintenance therapy in endometrial cancer in 1Q 2022.
If SIENDO data are positive, submit supplemental New Drug Application for selinexor in endometrial cancer to the U.S. Food & Drug Administration in 1H 2022.
Dose first patient in Phase 3 study evaluating selinexor, pomalidomide and dexamethasone in patients with relapsed or refractory multiple myeloma in 1Q 2022.
EMA decision expected in second-line multiple myeloma based on BOSTON study results in 1H 2022.
Realize further milestones and royalties from approvals and sales in ex-US territories in 2022.
The financial information presented in this press release may be adjusted as a result of the completion of customary quarterly and annual review and audit procedures.

Listen to the Webcast

These achievements and updates will be discussed during a webcast presentation at the 40th Annual J.P. Morgan Healthcare Conference to be held on Tuesday, January 11, 2022, at 3:45 p.m. ET, followed by a question-and-answer breakout session at 4:05 p.m. ET. A live webcast of the presentation and breakout session, along with accompanying slides, can be accessed under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived replay will be available for 30 days following the event. The presentation slides will also be available on the Company’s website following the event.

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in a growing number of ex-U.S. territories and countries, including Europe, the United Kingdom, China, South Korea and Israel, and is marketed in those areas by Karyopharm’s global partners. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer, myelodysplastic syndromes and myelofibrosis.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti–CD38 monoclonal antibody (Xd).
For the treatment of adult patients with relapsed or refractory diffuse large B–cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony–stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo–Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3–4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3–4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.

Selecta Biosciences and Ginkgo Bioworks Announce New Collaboration to Develop Next-Generation Gene Therapy Capsids

On January 10, 2022 Selecta Biosciences, Inc. (Nasdaq: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, and Ginkgo Bioworks (NYSE: DNA), the leading horizontal platform for cell programming, reported a collaboration to develop safer and more effective next-generation gene therapy viral capsids (Press release, Selecta Biosciences, JAN 10, 2022, View Source [SID1234598506]). This partnership leverages the unique platforms of both companies as they seek to develop next-generation viral capsids with improved transduction, enhanced tissue tropism and reduced immunogenicity. Ginkgo plans to design and engineer the capsids and Selecta will conduct all pre-clinical and clinical studies thereafter.

"We are excited to continue our partnership with Ginkgo in a second collaboration that leverages Ginkgo’s leading cell engineering platform to design viral vector capsids with the goal of improved transduction, enhanced tissue tropism and reduced immunogenicity," said Carsten Brunn, CEO of Selecta Biosciences. "By combining ImmTOR with next-generation, fit-for-purpose AAV vectors, we hope to further improve the safety and efficacy of AAV-mediated gene therapies."

Jason Kelly, CEO of Ginkgo Bioworks, added, "Gene therapies utilize naturally occurring viral capsids that elicit immune responses and often exhibit safety and toxicity issues. We believe our collaboration with Selecta has the potential to confront major challenges within the gene therapy space and represents an important application of our cell programming platform. We look forward to executing on our shared vision and offering a potential solution for challenges in gene therapies in order to enable treatment for millions of patients."

Under the terms of the collaboration, Ginkgo is eligible to earn upfront research and development fees and milestone payments, including certain milestone payments in the form of Selecta common stock. In addition, Ginkgo is eligible to earn clinical, regulatory and commercial milestone payments of up to $200 million in cash for each of a specified number of products, which have the potential to total, in the aggregate, up to $1.1 billion. Ginkgo is also entitled to potential further downstream value in the form of royalties on sales.

Deciphera Pharmaceuticals Provides Corporate Update and Highlights Key 2022 Corporate Milestones

On January 10, 2022 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a commercial-stage biopharmaceutical company developing innovative medicines to improve the lives of people with cancer, reported a corporate update and highlighted key 2022 milestones in conjunction with its presentation at the 40th Annual J.P. Morgan Healthcare Conference (Press release, Deciphera Pharmaceuticals, JAN 10, 2022, View Source [SID1234598505]). The Company will webcast its presentation today at 9:00 AM ET at View Source

"Deciphera is well-positioned as we enter 2022 with multiple high-value clinical programs and a successful commercial franchise in QINLOCK, all generated by our proven and productive research engine," said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. "We are excited about our leading portfolio of novel kinase inhibitors, including our first-in-class autophagy pathway inhibitor, DCC-3116, and our newly disclosed pan-RAF research program, both of which seek to address the challenges of drug resistance in patients with cancer. We believe that DCC-3116, designed to inhibit the autophagy pathway by inhibiting the ULK kinase, has the potential to address a broad range of human cancers, and planning is now underway to expand this development program to include a KRAS G12C inhibitor combination in non-small cell lung cancer (NSCLC). We plan to present initial data from the single agent dose escalation portion of this Phase 1 study later this year. We also announced today our plans to nominate a new clinical development candidate from our pan-RAF research program later this year, which we believe has the potential to be a best-in-class agent that can address unmet medical needs as a single agent and in combination."

Mr. Hoerter continued, "We kicked off 2022 with the launch of QINLOCK in Germany and we plan to transition to a post-approval paid access program in France in the first half of this year. Our existing QINLOCK commercial footprint with sarcoma specialists also serves as an excellent foundation for the future commercialization of our potential best-in-class CSF1R inhibitor, vimseltinib. The Phase 3 MOTION study of vimseltinib in tenosynovial giant cell tumor (TGCT) is currently underway and we expect to present updated data from our Phase 1/2 study in patients with TGCT later this year."

In 2022, the Company seeks to achieve the following milestones:

QINLOCK (ripretinib)

Execute on the commercial launch of QINLOCK in fourth-line GIST in Germany. Launch in Germany is ongoing as of January 1, 2022.
Transition to post-approval paid access program in France in the first half of 2022.
Present results of the Phase 3 INTRIGUE study in second-line GIST at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Plenary Series Session on January 25, 2022.
Vimseltinib

Enrollment underway in the Phase 3 MOTION study of vimseltinib, an orally administered, potent, and highly selective switch-control kinase inhibitor of CSF1R, for the treatment of TGCT.
Present updated data from the Phase 1/2 study in TGCT patients in the second half of 2022.
DCC-3116

Present data from the single agent dose escalation portion of this Phase 1 study of DCC-3116, a first-in-class ULK kinase inhibitor designed to inhibit autophagy for the treatment of patients with advanced or metastatic tumors with a mutant a RAS or RAF gene, in the second half of 2022. DCC-3116 is currently being investigated as a single agent and in combination.
Initiate Phase 1 study dose expansion in the second half of 2022 in combination with trametinib, a U.S. Food and Drug Administration-approved MEK inhibitor, in patients with selected mutations in advanced or metastatic pancreatic ductal adenocarcinoma, NSCLC, colorectal cancer, and melanoma.
Planning underway to add a combination with a KRAS G12C inhibitor in NSCLC to the ongoing Phase 1 study, subject to feedback from regulatory authorities.
Present additional preclinical data for DCC-3116 in 2022 and continue to explore preclinical combinations of DCC-3116 with multiple additional targeted oncology agents with diverse mechanisms of action.
Proprietary Drug Discovery Platform

Nominate a development candidate in 2022 from the pan-RAF inhibitor research program, discovered using the Company’s novel switch-control inhibitor platform.
Presentation at the 40th Annual J.P. Morgan Healthcare Conference

Deciphera will webcast its corporate presentation from the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 9:00 AM ET. A live webcast of the presentation can be accessed under "Events & Presentations" in the investors section of the Company’s website at deciphera.com. A replay of the webcast will be archived on the Company’s website for 90 days following the presentation. In conjunction with the conference, the Company has also updated its corporate presentation, which can be found here: View Source

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 40th Annual J.P. Morgan Healthcare Conference

On January 10, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported tthat it will present an update on its commercial progress and R&D pipeline at the 40th Annual J.P. Morgan Healthcare Conference today, Monday Jan. 10th at 7:30am EST (Press release, PTC Therapeutics, JAN 10, 2022, View Source [SID1234598504]). Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2021 accomplishments and highlight upcoming potential value-creating milestones. Preliminary 2021 unaudited financial results and 2022 financial guidance will also be provided. The presentation will be webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.

Key 2021 Corporate Highlights:

Unaudited net product revenue of $429 million in 2021 representing 29% year-over-year growth.
Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of $424 million for Translarna (ataluren) and Emflaza (deflazacort) in 2021. Cumulative net product revenue for Translarna exceeds $1 billion and Emflaza exceeds $500 million, since respective launches.
Translarna growth was driven by new patients and high compliance in existing geographies and continued geographic expansion.
Emflaza growth was due to continued new prescriptions, high compliance, less patient discontinuations and more favorable access.
Evrysdi (risdiplam) has shown continued rapid uptake in the United States and is now approved in all major markets including the European Union and Japan. Evrysdi is a product of the Spinal Muscular Atrophy (SMA) collaboration between PTC, the SMA Foundation and Roche.
Waylivra(volanesorsen), the only treatment for familial chylomicronemia syndrome was approved by Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária (ANVISA), and both Waylivra and Tegsedi(inotersen) received Category 1 classification from Câmara de Regulação do Mercado de Medicamentos – CMED (Drug Market Regulation Chamber) in Brazil. Category 1 classification is given to innovative treatments that provide greater efficacy than the standard of care and allows for pricing in line with international markets.
PTC successfully advanced its clinical pipeline in 2021:
APHENITY, a Phase 3 registration-directed trial of PTC923 in phenylketonuria (PKU) was initiated.
Phase 1 healthy volunteer trials of PTC518 and PTC857 were completed.
Advanced the oncology platform with the completion of Phase 1b studies of unesbulin in leiomyosarcoma and diffuse intrinsic pontine glioma (DIPG).
Completed enrollment for the vatiquinone MOVE-FA registration-directed trial with results expected in the second quarter of 2023.
2022 Potential Key Value-Creating Milestones:

Results from Study 041 for Translarna are expected mid-year 2022 and if positive could support
re-submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA).
Results for MIT-E, the registration-directed study of vatiquinone in mitochondrial disease associated seizures, are expected in the fourth quarter of 2022.
Results are expected by year end 2022 for the Phase 3 registration-directed study, APHENITY, for PTC923 in patients with PKU.
From the splicing platform, the PIVOT-HD Phase 2 study of PTC518 in Huntington’s disease patients is planned to initiate in the first quarter of 2022.
From the Bio-e platform, the registration-directed CardinALS study of PTC857 in amyotrophic lateral sclerosis (ALS) patients is expected to be initiated in the second quarter of 2022.
Progress in the gene therapy platform is anticipated in 2022:
PTC expects an opinion from the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for PTC-AADC, the first gene therapy for AADC deficiency, in April 2022.
Submission of a Biologics License Application (BLA) to the FDA for PTC-AADC is expected in the second quarter of 2022.
Preliminary Unaudited 2021 Financial Results:

Total unaudited net revenue for full year 2021 was approximately $536 million.
Total unaudited net product revenue for full year 2021 was approximately $429 million.
DMD franchise revenue for year end 2021 included net product revenue for Translarna of approximately $236 million, with $70 million in revenue in the fourth quarter, and Emflaza of approximately $188 million, with $48 million in revenue in the fourth quarter.
PTC expects to report approximately $107 million in 2021 collaboration and royalty revenue associated with Evrysdi.
PTC expects to report 2021 year-end cash, cash equivalents and marketable securities of approximately $773 million.
PTC is currently in the process of finalizing its financial results for the 2021 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2021, subject to the completion of PTC’s financial closing procedures.

2022 Financial Guidance:

PTC anticipates total revenues for the full year 2022 to be between $700 and $750 million.
PTC anticipates net product revenues for the DMD franchise for the full year 2022 to be between $475 and $495 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2022 to be between $915 and $965 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2022 to be between $800 and $850 million, excluding estimated non-cash, stock-based compensation expense of $115 million.
Non-GAAP Financial Measures:

In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.